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Thrombocytopenia 7(THC7)

MedGen UID:
1768257
Concept ID:
C5436874
Disease or Syndrome
Synonym: THROMBOCYTOPENIA, AUTOSOMAL DOMINANT, 7
 
Gene (location): IKZF5 (10q26.13)
 
Monarch Initiative: MONDO:0030867
OMIM®: 619130

Definition

Thrombocytopenia-7 (THC7) is an autosomal dominant disorder characterized by reduced peripheral platelet count. The expression and severity of the disorder is highly variable: some patients have no bleeding symptoms, whereas other have recurrent petechiae, epistaxis, or more severe bleeding episodes. A common finding is decreased alpha-granules in the platelets. There are variable findings on light and electron microscopic analysis: some patients have normal platelet morphology, whereas others show abnormal platelet morphology with cytoskeletal defects. Flow cytometric studies may show reduced expression of platelet membrane glycoproteins and activation markers (summary by Lentaigne et al., 2019 and Leinoe et al., 2021). For a general phenotypic description and a discussion of genetic heterogeneity of thrombocytopenia, see 313900. [from OMIM]

Clinical features

From HPO
Post-partum hemorrhage
MedGen UID:
18592
Concept ID:
C0032797
Pathologic Function
Significant maternal hemorrhage/blood loss following deilvery of a child.
Thrombocytopenia
MedGen UID:
52737
Concept ID:
C0040034
Disease or Syndrome
A reduction in the number of circulating thrombocytes.
Impaired ristocetin-induced platelet aggregation
MedGen UID:
868749
Concept ID:
C4023154
Pathologic Function
Abnormal response to ristocetin as manifested by reduced or lacking aggregation of platelets upon addition of ristocetin.
Impaired arachidonic acid-induced platelet aggregation
MedGen UID:
868750
Concept ID:
C4023155
Pathologic Function
Abnormal response to arachidonic acid as manifested by reduced or lacking aggregation of platelets upon addition of arachidonic acid.
Impaired collagen-induced platelet aggregation
MedGen UID:
870264
Concept ID:
C4024703
Finding
Abnormal response to collagen or collagen-mimetics as manifested by reduced or lacking aggregation of platelets upon addition collagen or collagen-mimetics.
Impaired ADP-induced platelet aggregation
MedGen UID:
870824
Concept ID:
C4025282
Finding
Abnormal platelet response to ADP as manifested by reduced or lacking aggregation of platelets upon addition of ADP.
Reduced platelet dense granules
MedGen UID:
1785275
Concept ID:
C5539663
Finding
Decreased number of platelet dense granules, a type of platelet organelle.
Reduced platelet alpha granules
MedGen UID:
1789060
Concept ID:
C5539664
Finding
A reduced number of platelet alpha granules.

Professional guidelines

PubMed

Demirci NS, Aksoy S, Özdemir NY, Erdem GU, Ozcelik M, Tanrikulu E, Eren T, Bozkaya Y, Sahin S, Başol F, Aslan SA, Zengin N, Güllü İ
Curr Med Res Opin 2017 Mar;33(3):401-407. Epub 2016 Dec 8 doi: 10.1080/03007995.2016.1257984. PMID: 27817239
Coelho RC, Reinert T, Campos F, Peixoto FA, de Andrade CA, Castro T, Herchenhorn D
Int Braz J Urol 2016 Jul-Aug;42(4):694-703. doi: 10.1590/S1677-5538.IBJU.2015.0226. PMID: 27564279Free PMC Article
De la Cruz Vicente F, Carrillo-Cruz E, Rodriguez MS, Marín Niebla A, Galiana ML, Gonzalez JF, Cuadrado IM, Campos JG, Tocino IE, Rios-Herranz E, Perez-Simón JA
Eur J Haematol 2014 Dec;93(6):469-75. Epub 2014 Jul 2 doi: 10.1111/ejh.12384. PMID: 24853381

Recent clinical studies

Etiology

Touzeau C, Perrot A, Hulin C, Manier S, Macro M, Chretien ML, Karlin L, Escoffre M, Jacquet C, Tiab M, Leleu X, Avet-Loiseau H, Jobert A, Planche L, Corre J, Moreau P
Blood 2024 May 16;143(20):2029-2036. doi: 10.1182/blood.2023023597. PMID: 38394666
Vergote I, Pérez-Fidalgo JA, Hamilton EP, Valabrega G, Van Gorp T, Sehouli J, Cibula D, Levy T, Welch S, Richardson DL, Guerra EM, Scambia G, Henry S, Wimberger P, Miller DS, Klat J, Martínez-Garcia J, Raspagliesi F, Pothuri B, Romero I, Bergamini A, Slomovitz B, Schochter F, Høgdall E, Fariñas-Madrid L, Monk BJ, Michel D, Kauffman MG, Shacham S, Mirza MR, Makker V; ENGOT-EN5/GOG-3055/SIENDO Investigators
J Clin Oncol 2023 Dec 10;41(35):5400-5410. Epub 2023 Sep 5 doi: 10.1200/JCO.22.02906. PMID: 37669480
O'Connor OA, Horwitz S, Masszi T, Van Hoof A, Brown P, Doorduijn J, Hess G, Jurczak W, Knoblauch P, Chawla S, Bhat G, Choi MR, Walewski J, Savage K, Foss F, Allen LF, Shustov A
J Clin Oncol 2015 Aug 10;33(23):2492-9. Epub 2015 Jun 22 doi: 10.1200/JCO.2014.59.2782. PMID: 26101246Free PMC Article
Sharif S, Zahid I, Routledge T, Scarci M
Interact Cardiovasc Thorac Surg 2011 Jun;12(6):1040-5. Epub 2011 Mar 8 doi: 10.1510/icvts.2010.256289. PMID: 21388982
O'Brien ME, Ciuleanu TE, Tsekov H, Shparyk Y, Cuceviá B, Juhasz G, Thatcher N, Ross GA, Dane GC, Crofts T
J Clin Oncol 2006 Dec 1;24(34):5441-7. doi: 10.1200/JCO.2006.06.5821. PMID: 17135646

Diagnosis

Touzeau C, Perrot A, Hulin C, Manier S, Macro M, Chretien ML, Karlin L, Escoffre M, Jacquet C, Tiab M, Leleu X, Avet-Loiseau H, Jobert A, Planche L, Corre J, Moreau P
Blood 2024 May 16;143(20):2029-2036. doi: 10.1182/blood.2023023597. PMID: 38394666
Shen X, Huang Y, Guo H, Peng H, Yao S, Zhou M, Liu H, Lin HC, Zhou P
Clin Res Hepatol Gastroenterol 2021 Mar;45(2):101495. Epub 2020 Jul 25 doi: 10.1016/j.clinre.2020.06.019. PMID: 32723673
Wangrangsimakul T, Greer RC, Chanta C, Nedsuwan S, Blacksell SD, Day NPJ, Paris DH
J Pediatric Infect Dis Soc 2020 Apr 30;9(2):202-209. doi: 10.1093/jpids/piz014. PMID: 30864670Free PMC Article
Paludo J, Abeykoon JP, Kumar S, Shreders A, Ailawadhi S, Gertz MA, Kourelis T, King RL, Reeder CB, Leung N, Kyle RA, Buadi FK, Habermann TM, Dingli D, Witzig TE, Dispenzieri A, Lacy MQ, Go RS, Lin Y, Gonsalves WI, Warsame R, Lust JA, Rajkumar SV, Ansell SM, Kapoor P
Br J Haematol 2017 Oct;179(1):98-105. Epub 2017 Aug 8 doi: 10.1111/bjh.14826. PMID: 28786474
Jain R, Chartash E, Susin M, Furie R
Semin Arthritis Rheum 1994 Dec;24(3):173-82. doi: 10.1016/0049-0172(94)90073-6. PMID: 7899875

Therapy

Touzeau C, Perrot A, Hulin C, Manier S, Macro M, Chretien ML, Karlin L, Escoffre M, Jacquet C, Tiab M, Leleu X, Avet-Loiseau H, Jobert A, Planche L, Corre J, Moreau P
Blood 2024 May 16;143(20):2029-2036. doi: 10.1182/blood.2023023597. PMID: 38394666
Vergote I, Pérez-Fidalgo JA, Hamilton EP, Valabrega G, Van Gorp T, Sehouli J, Cibula D, Levy T, Welch S, Richardson DL, Guerra EM, Scambia G, Henry S, Wimberger P, Miller DS, Klat J, Martínez-Garcia J, Raspagliesi F, Pothuri B, Romero I, Bergamini A, Slomovitz B, Schochter F, Høgdall E, Fariñas-Madrid L, Monk BJ, Michel D, Kauffman MG, Shacham S, Mirza MR, Makker V; ENGOT-EN5/GOG-3055/SIENDO Investigators
J Clin Oncol 2023 Dec 10;41(35):5400-5410. Epub 2023 Sep 5 doi: 10.1200/JCO.22.02906. PMID: 37669480
O'Connor OA, Horwitz S, Masszi T, Van Hoof A, Brown P, Doorduijn J, Hess G, Jurczak W, Knoblauch P, Chawla S, Bhat G, Choi MR, Walewski J, Savage K, Foss F, Allen LF, Shustov A
J Clin Oncol 2015 Aug 10;33(23):2492-9. Epub 2015 Jun 22 doi: 10.1200/JCO.2014.59.2782. PMID: 26101246Free PMC Article
Sharif S, Zahid I, Routledge T, Scarci M
Interact Cardiovasc Thorac Surg 2011 Jun;12(6):1040-5. Epub 2011 Mar 8 doi: 10.1510/icvts.2010.256289. PMID: 21388982
O'Brien ME, Ciuleanu TE, Tsekov H, Shparyk Y, Cuceviá B, Juhasz G, Thatcher N, Ross GA, Dane GC, Crofts T
J Clin Oncol 2006 Dec 1;24(34):5441-7. doi: 10.1200/JCO.2006.06.5821. PMID: 17135646

Prognosis

Stein EM, DiNardo CD, Fathi AT, Pollyea DA, Stone RM, Altman JK, Roboz GJ, Patel MR, Collins R, Flinn IW, Sekeres MA, Stein AS, Kantarjian HM, Levine RL, Vyas P, MacBeth KJ, Tosolini A, VanOostendorp J, Xu Q, Gupta I, Lila T, Risueno A, Yen KE, Wu B, Attar EC, Tallman MS, de Botton S
Blood 2019 Feb 14;133(7):676-687. Epub 2018 Dec 3 doi: 10.1182/blood-2018-08-869008. PMID: 30510081Free PMC Article
O'Connor OA, Horwitz S, Masszi T, Van Hoof A, Brown P, Doorduijn J, Hess G, Jurczak W, Knoblauch P, Chawla S, Bhat G, Choi MR, Walewski J, Savage K, Foss F, Allen LF, Shustov A
J Clin Oncol 2015 Aug 10;33(23):2492-9. Epub 2015 Jun 22 doi: 10.1200/JCO.2014.59.2782. PMID: 26101246Free PMC Article
Sharif S, Zahid I, Routledge T, Scarci M
Interact Cardiovasc Thorac Surg 2011 Jun;12(6):1040-5. Epub 2011 Mar 8 doi: 10.1510/icvts.2010.256289. PMID: 21388982
O'Brien ME, Ciuleanu TE, Tsekov H, Shparyk Y, Cuceviá B, Juhasz G, Thatcher N, Ross GA, Dane GC, Crofts T
J Clin Oncol 2006 Dec 1;24(34):5441-7. doi: 10.1200/JCO.2006.06.5821. PMID: 17135646
Mantadakis E, Danilatou V, Stiakaki E, Kalmanti M
Am J Hematol 2004 Nov;77(3):303-10. doi: 10.1002/ajh.20180. PMID: 15495242

Clinical prediction guides

Twu CW, Lin PJ, Tsou HH, Liu YC, Jiang RS, Liang KL, Lin TY, Wang WY, Lin JC
Head Neck 2022 Jun;44(6):1453-1461. Epub 2022 Apr 1 doi: 10.1002/hed.27044. PMID: 35362634
Qi F, Wang WH, He XH, Chen B, Gui L, Fang H, Liu P, Wang SL, Yang JL, Song YW, Yang S, Qi SN, Zhou SY, Li YX, Dong M
Int J Radiat Oncol Biol Phys 2018 Sep 1;102(1):61-70. Epub 2018 May 29 doi: 10.1016/j.ijrobp.2018.05.046. PMID: 30102205
Lim KH, Lee HY, Song SY
Chin Med J (Engl) 2013;126(24):4644-8. PMID: 24342304
Steinberg M
Clin Ther 2008 Sep;30(9):1590-617. doi: 10.1016/j.clinthera.2008.09.015. PMID: 18840366
Saracino B, Arcangeli G, Mecozzi A, Tirindelli Danesi D, Cruciani E, Altavista P, Giannarelli D
Clin Ter 1998 May-Jun;149(3):183-9. PMID: 9842100

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